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Apellis' rare blood disorder drug improves hemoglobin count in late-stage study

Apellis Pharmaceuticals Inc. said its drug APL-2 increased hemoglobin protein in patients with a rare life-threatening disease that destroys red blood cells during a late-stage study.

The share price of the Crestwood, Ky.-based biotech was up 20.6% to $36.42 as of 11:11 a.m. ET on Jan. 7.

Apellis was comparing APL-2 with eculizumab, sold by Alexion Pharmaceuticals Inc. as Soliris, as a treatment for patients with paroxysmal nocturnal hemoglobinuria in the head-to-head phase 3 trial called Pegasus.

The disorder is caused by mutations in a gene in bone marrow stem cell that results in the production of red blood cells, which lack certain proteins meant for protecting the cells. The protein deficiency is responsible for most issues related to paroxysmal nocturnal hemoglobinuria, or PNH, that include blood clots and infection.

Patients receiving twice-weekly injections of APL-2, or pegcetacoplan, had higher average hemoglobin after 16 weeks, while patients treated with eculizumab recorded a decrease since the start of the trial, according to the company.

APL-2 and eculizumab were equally effective in keeping patients transfusion free and managing the number of immature red blood cells, Apellis said in its Jan. 7 press release.

Apellis Chief Medical Officer Federico Grossi said the company expects to meet with regulators in the first half of the year to discuss the next steps.

Leerink analysts Joseph Schwartz and Dae Gon Ha noted that APL-2 as a treatment for PNH may become Apellis' first commercial opportunity. However, the drug faces tough competition as the strong adoption of Soliris and rapid ongoing conversion of patients to Alexion's Ultomiris that was approved by the U.S. Food and Drug Administration to treat PNH in December 2018.

The FDA has granted fast-track designation to APL-2 for PNH. The U.S. agency has also granted APL-2 orphan-drug designation to treat patients with autoimmune hemolytic anemia, which activates certain parts of the immune system, resulting in the premature destruction of red blood cells, and C3 glomerulopathy, a group of rare renal disorders that impacts the kidneys' ability to filter blood.

Apellis will face strong competition from Sanofi's sutimlimab if it expands APL-2 to treat cold agglutinin disease, or CAD, Leerink's analysts wrote.

Apellis is studying APL-2 during a phase 2 trial in patients with CAD.